Clinical Outcomes and Disease Management for Patients with Paroxysmal Nocturnal Haemoglobinuria in China: Results from a Real-World Study.

Background: Paroxysmal nocturnal haemoglobinuria (PNH) is a chronic haematological disorder caused by uncontrolled complement activation, leading to intravascular haemolysis, indicated by increased lactate dehydrogenase levels (LDH). Standard-of-care complement factor 5 inhibitors have only recently been approved in China. Using real-world data, this study aimed to understand treatment pattern and burden of disease, including thrombosis risk, for Chinese patients with PNH.

Patient-reported outcomes and daily activity assessed with a digital wearable device in patients with paroxysmal nocturnal hemoglobinuria treated with ravulizumab: REVEAL, a prospective, observational study.

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic blood disorder. Symptoms such as fatigue can have a substantial impact on patients' physical activity levels, sleep, quality of life, and work productivity. Ravulizumab treatment can reduce thrombosis risk, improve survival and quality of life, and reduce fatigue in PNH, but information is limited on how it impacts sleep and physical activity.

No impact of Asian ethnicity on EORTC QLQ-C30 scores: Group differences and differential item functioning in paroxysmal nocturnal hemoglobinuria.

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening terminal-complement-mediated disease resulting in intravascular hemolysis and thrombosis with significant morbidity and premature mortality. There exists no disease-specific quality-of-life (QOL) measure for PNH. Its QOL effects resemble those of hematologic cancers, which supports the use of cancer-specific QOL measures in PNH clinical trials.

Indirect and Direct Mapping of the Cancer-Specific EORTC QLQ-C30 onto EQ-5D-5L Utility Scores.

Objective: The aim of this study was to develop a response mapping algorithm to predict EQ-5D-5L utilities from European Organisation for Research and Treatment Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) scores and compare performance with direct mapping approaches to identify the best performing algorithm.

Methods: The Multi-Instrument Comparison dataset contains responses to both the EQ-5D-5L and QLQ-C30 questionnaires from 692 individuals with a broad range of cancers. Response mapping was conducted, fitting ordered logistic regressions to predict response levels for each of the five EQ-5D dimensions and utilities were predicted using the US and Japanese EQ-5D-5L value sets to test the algorithm performance.

Norm-based comparison of the quality-of-life impact of ravulizumab and eculizumab in paroxysmal nocturnal hemoglobinuria.

Aims: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening intravascular hematologic disorder with significant morbidity and premature mortality. Clinical trials (NCT02946463 and NCT03056040) comparing ravulizumab with eculizumab for PNH have supported the non-inferiority of the former and similar safety and tolerability. This secondary analysis compared PNH trial participants after 26 weeks on either treatment (n = 438) to a general-population sample (GenPop) (n = 15,386) and investigated response-shift effects.

A US cost-minimization model comparing ravulizumab versus eculizumab for the treatment of atypical hemolytic uremic syndrome.

Aims: Ravulizumab, engineered from eculizumab, provides sustained C5 inhibition in atypical hemolytic uremic syndrome (aHUS) while reducing dosing frequency (every 8 vs 2 weeks, respectively). Treatment choice often carries significant financial implications. This study compared the economic consequences of ravulizumab and eculizumab for treating aHUS.

Cost-utility analysis of life-long prophylaxis with recombinant factor VIIIFc vs recombinant factor VIII for the management of severe hemophilia A in Sweden.

Aims: Prophylaxis with recombinant factor VIII (rFVIII) is the standard of care for severe hemophilia A in Sweden. The need for frequent injections with existing rFVIII products may, however, result in poor adherence to prophylaxis, leading to increased bleeding and long-term joint damage. Recombinant FVIIIFc (rFVIIIFc) is an extended half-life fusion protein which can offer prolonged protection and reduced dosing frequency.

Continuous prophylaxis with recombinant factor IX Fc fusion protein and conventional recombinant factor IX products: comparisons of efficacy and weekly factor consumption.

Background: Continuous prophylaxis for patients with hemophilia B requires frequent injections that are burdensome and that may lead to suboptimal adherence and outcomes. Hence, therapies requiring less-frequent injections are needed. In the absence of head-to-head comparisons, this study compared the first extended-half-life-recombinant factor IX (rFIX) product-recombinant factor IX Fc fusion protein (rFIXFc)-with conventional rFIX products based on annualized bleed rates (ABRs) and factor consumption reported in studies of continuous prophylaxis.

Health economic modeling of the potential cost saving effects of Neurally Adjusted Ventilator Assist.

Objectives: Asynchrony between patient and ventilator breaths is associated with increased duration of mechanical ventilation (MV). Neurally Adjusted Ventilatory Assist (NAVA) controls MV through an esophageal reading of diaphragm electrical activity via a nasogastric tube mounted with electrode rings. NAVA has been shown to decrease asynchrony in comparison to pressure support ventilation (PSV).

Atomoxetine's effect on societal costs in Sweden.

Objective: To compare societal costs between patients treated with atomoxetine and placebo in Sweden.

Method: Ninety-nine pediatric ADHD patients were randomized to a 10-week double-blind treatment with atomoxetine (n = 49) or placebo (n = 50). All parents received four sessions of psycho-education.

Long-term psychosocial and health economy consequences of ADHD, autism, and reading-writing disorder: a prospective service evaluation project.

Objective: The study aims to evaluate psychosocial, societal, and family cost consequences of a psychoeducational intervention program.

Methods: Sixty boys with ADHD, Asperger syndrome/high-functioning autism (AS/HFA), and reading and writing disorder (RD/WD) were allocated to participate in a service evaluation project. Every other boy in each diagnostic group was randomly allocated to receive either (a) a special education program (clinical index group) or (b) follow-up without the special education program (clinical comparison group).